N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide (hereinafter “Compound 1”) has the structure below:
Compound 1 is described and claimed in International PCT publication WO 2006002421 and has the following molecular formula: C24H28N2O3.
Compound 1 has been demonstrated to restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, the defective cell membrane protein responsible for the progression of CF. Defects in the CFTR protein can affect the transport of chloride and other ions across cells, and lead to the accumulation of thick, sticky mucus in the lungs of patients with CF. This mucus can foster chronic infection and inflammation, and can result in irreversible lung damage. Potentiator compounds such as Compound 1 can increase the probability that the CFTR channel is open, which could result in an increase in chloride transport across the cell surface in some patients. In laboratory experiments, using cells from patients with CF where CFTR proteins are present on the cell surface, Compound 1 has restored the function of defective CFTR channels.